巨核细胞发育和血小板生成的调控机理及巨核细胞和血小板的临床应用

Regulation mechanisms of megakaryopoiesis and platelet formation and its clinical application

血小板是血液中行使凝血和止血功能的重要成分,近年来研究发现血小板还有日益增多的各种功能。血小板的数量和功能缺陷本身可导致某些疾病,而某些疾病中出现的血小板数量和功能缺陷,也严重影响疾病的预后。自1906年首次确认血小板来源于巨核细胞后,血小板的各种病理改变就必然与巨核细胞的发育成熟过程密切相关。本文拟对造血系统中巨核细胞系列的发育成熟和血小板生成释放的过程及其调控机理,以及在多种疾病中针对巨核细胞、血小板的靶向干预策略做一简明扼要的综述,使读者获得此领域比较全面综合的理解。

Platelets, as the major effector of coagulation and hemostasis, are generated by mature megakaryocytes which differentiated from the committed erythroid/megakaryoid progenitors. Megakaryopoiesis and platelet production are genetically and epigenetically regulated by transcription factors and a number of stimulating signal pathways through some critical steps. The megakaryocytes and platelets have their distinct phenotype and functions. The mutation or deletion of genes encoding transcription factor or signaling proteins results in abnormalities in amount and function of platelets, which may lead to familiar inherited platelet disorders with propensity to myeloid malignancies, including myelodysplastic syndrome/bone marrow hyperplastic tumor. Currently, megakaryocyte and platelet are increasingly applied as target or vesicle in targeting therapeutic strategies.